A mother's love drives her to pursue experimental treatment as two of her four children battle a rare genetic disease that ...

The EMA’s positive opinion on Kygevvi marks a turning point for patients with TK2d, an ultra-rare genetic muscle disease previously managed only with supportive care.

A phase 2a trial of NMD Pharma’s rare disease prospect ignaseclant has missed its primary endpoint. | A phase 2a trial of NMD ...

Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, ...

Researchers at Kumamoto University have demonstrated that iron supplementation can significantly alleviate muscle pathology ...

Congenital muscular dystrophies (CMDs) comprise a heterogeneous group of inherited disorders primarily affecting muscle integrity and function, often with accompanying brain and ocular abnormalities.

When it comes to muscular diseases, most of us have heard of especially common ones like muscular dystrophy and Lou Gehrig's disease. But one of the rarest muscular disorders is also one of the most ...

A team at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) has developed an innovative method known as TEVs-TTN, for studying the specific mechanical functions of proteins through their ...

Researchers in public health have reported in the first broad study in the United States the frequency of two muscle-weakness disorders that strike mostly boys: Duchenne muscular dystrophy and Becker ...

You're relaxing on the sofa when suddenly your eyelid starts twitching. Or perhaps it's a muscle in your arm, your leg, or ...